FDA approves new targeted therapy for CLL11 Apr 2016
The FDA approved the B-cell lymphoma (BCL)-2 inhibitor venetoclax (Venclexta) for patients with previously treated chronic lymphocytic leukemia (CLL) associated with 17-p deletion. A key mediator of apoptosis, BCL-2 overexpression occurs in several malignancies, resulting in an anti-apoptotic effect. The FDA action represents the first-ever approval of a BCL-2 targeting drug.
The 17-p deletion occurs in about 10% of newly diagnosed chronic lymphocytic leukemia and is associated with treatment resistance. The proportion of affected patients increases to about 20% in relapsed CLL.
Venetoclax led to an objective response rate of 80%
"These patients now have a new targeted therapy that inhibits a protein involved in keeping tumor cells alive," Richard Pazdur, MD, director of the FDA Office of Hematology and Oncology Products, commented. "For certain patients with CLL who have not had favorable outcomes with other therapies, Venclexta may provide a new option for their specific condition."
The principal supporting clinical evidence for the approval application came from a single-arm phase 2 trial involving 106 patients with 17-p deletion CLL that had progressed or relapsed after first-line treatment. Daily oral therapy with increasing doses of venetoclax led to an objective response rate of 80%, including complete and partial remission.
Drug’s common adverse events
The agent worked so quickly and so effectively that investigators found it necessary to take precautions to prevent tumor lysis syndrome. Most responding patients had minimal residual disease status at 12 months.
The most common grade 3/4 adverse events in the phase 2 trial were neutropenia in 40% of patients, anemia in 18%, and thrombocytopenia in 15%. According to the FDA, common adverse events associated with venetoclax include diarrhea, nausea, upper respiratory tract infection, and fatigue. The drug is manufactured by AbbVie and marketed by AbbVie and Genentech.Source: MedPage Today